The PKD Foundation has announced the U.S. Food and Drug Administration (FDA) granted approval of Jynarque to be the first treatment in the United States for adult patients with rapidly progressing autosomal dominant polycystic kidney disease (ADPKD), the most common form of polycystic kidney disease (PKD). The PKD Foundation not only supported early studies that led to the development of Jynarque as a treatment, but also helped guide PKD patients to the Jynarque clinical trials. Jynarque was developed by Otsuka Pharmaceuticals.
PKD, a chronic, genetic disease, is characterized by uncontrolled growth of cysts in the kidneys and other organs and can lead to kidney failure. Previously, there has been no treatment specifically for PKD in the U.S. The only treatment options were to receive a transplant or begin dialysis. And though there is no cure, patients who are prescribed Jynarque for PKD will see a slower decline in their kidney function, leading to improved health and well-being.
"Today is an historic day in providing hope to patients with polycystic kidney disease, and we are thrilled to be a part of this first milestone to treat patients with ADPKD," said Andy Betts, CEO of the PKD Foundation. "For the past 35 years, our goal has been to support PKD patients from care to cure. It is gratifying to play a part in the discovery of this treatment and to see it come to fruition."
Following this approval by the FDA, Jynarque will be available in the United States for patients in the coming weeks.
Jynarque slows the growth of cysts and the progression of the disease and has been shown to preserve kidney function. The treatment for ADPKD is an oral pill taken twice a day. It is a long-term use drug – extending the time before a patient needs dialysis or a transplant, though not curing the disease.
The efficacy of Jynarque in ADPKD was based on data from two of the largest Phase 3 clinical trials in patients with ADPKD. Lasting one and three years respectively, they led to Jinarc/Jynarque to be approved for adult patients with ADPKD in Japan, the European Union, Canada, South Korea, Switzerland, Hong Kong, Australia, Turkey, Taiwan and finally today here in the United States.